Gene Silencing Gene Therapy Genetic Disease Genomics 
XTalks
FEBRUARY 14, 2022
The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.
XTalks
MAY 4, 2021
The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Nearly one third of human genes lack CGIs, which would limit the use of the tool. Epigenetic Editing with CRISPR.
XTalks
MAY 4, 2021
The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Nearly one third of human genes lack CGIs, which would limit the use of the tool. Epigenome Editing with CRISPR.
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